Transforming Medicine in 'Cellicon Valley'

Dr. Carl June works with frozen CAR T cells in the Center for Cellular Immunotherapies at the Abramson Cancer Center of the University of Pennsylvania. (Photo/Penn Medicine)

These are exciting times in “Cellicon Valley,” better known as the Greater Philadelphia area. With renowned academic institutions and world-class hospitals, the region has long been recognized as a center of medical excellence.

Greater Philadelphia and Pennsylvania are also known for outstanding research, as evidenced by two academic research institutions which received nearly $1 billion in research grants from the U.S. National Institutes of Health (NIH) in 2017. The University of Pennsylvania and University of Pittsburgh were among the top five recipients of NIH funding in 2017, receiving more than $493 million and $485 million, respectively.

Philadelphia may also have the greatest concentration of foundational research in gene and cell therapy in the world. As far back as 1959, researchers from Fox Chase Cancer Center and University of Pennsylvania identified the Philadelphia chromosome, which contains an abnormality associated with chronic myelogenous leukemia.

Christopher P. Molineaux, President and CEO of Life Sciences Pennsylvania (LSPA), the statewide trade association that fosters life science growth and success, says Philadelphia’s evolution as a biotech hub is due to a combination of scientific and business expertise. “Our greatest asset is people and talent,” Molineaux says.

The head of LSPA knows what he’s talking about. In recent years the laboratories of Philadelphia’s universities and teaching hospitals have come to boast an impressive record of breakthrough discoveries – many involving gene and cell therapy.

In fact, the world’s first genetically engineered immune therapy is an example of a homegrown breakthrough. Developed by Dr. Carl June at the University of Pennsylvania’s Perelman School of Medicine, the therapy reprograms the body’s own immune cells to seek and destroy acute lymphoblastic leukemia cells.

The magnitude of June’s achievement was recognized by U.S. Food & Drug Administration Commissioner Scott Gottlieb, M.D. in a news release in August 2017. “We’re entering a new frontier in medical innovation,” Gottlieb said, “With the ability to reprogram a patient’s own cells to attack a deadly cancer. New technologies such as gene and cell therapies hold out the potential to transform medicine.”

There are currently more than 700 gene therapies at different stages of clinical development. However, taking a new drug or technology from laboratory to clinic requires additional expertise. A viable therapy isn’t just one that can be approved, but that can be commercialized. This involves finding sustainable pricing models and dealing with the complexities of logistical challenges.

Global biotechnology leader CSL Behring has its operational headquarters in King of Prussia, Pa., in the greater Philadelphia region. The company is developing a gene therapy candidate based on hematopoietic stem cells (HSC), or those that generate all the blood cells in an organism, to treat Sickle Cell Disease.

Therapies such as HSC gene therapygenetically modify an area of a person’s gene sequence that is missing or that negatively affects his or her health. Stem cells are removed, genetically altered and infused back into the patient.

The altered cells deliver what CSL Behring’s Head of Commercial Development Hematology, Jens Oltrogge, describes as “a molecular payload with the potential to liberate patients from a lifetime of maintenance by medicine.” Oltrogge adds that as genetic engineering and cell biology progress and new treatments and cures are discovered, gene therapy will play a role in changing the treatment paradigm.

“We are already seeing a shift in the focus of medicine from the disease to the patient, and from therapies that treat symptoms to regenerative and curative treatments,” Oltrogge says.

Today, there are 2,800 biotech companies in Pennsylvania and as far as Molineaux is concerned, “Cell and gene technology is a Philadelphia thing.” He may be right, but only time will tell whether the name “Cellicon Valley” sticks. One thing is certain – these are exciting times indeed for cell and gene therapy research in the greater Philadelphia region.